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Clinical Trials

Federal Right to Try

So it looks like a Right-to-Try bill is going to be signed into law. People who have been advocating this for years will now get a chance to see how it works out in practice – and in fact, I would encourage them to go ahead and put down some predictions about what they think might happen. Predicting that not much will change, or that it will be hard to notice the effects, etc., is not (to my mind) a particularly good answer for supporters of this approach, because one then wonders why it was worth going to the trouble at all. But I’m not a supporter, so that’s my prediction: little change, with potential for harm.

I’ve written about this issue many times before, but in case my views aren’t clear, here we go: I think Right to Try laws are largely useless, and have the potential to cause actual harm disproportionate to the good they might do. I think that there are sincere, principled advocates for them, but at the same time, there are many people on that side of the issue who are either ill-informed about medical research or who are simply grandstanding for votes and popular support.

In short, my biggest reasons for thinking this way are: (1) most investigational drugs don’t work. The failure rate in human clinical trials is around 90 per cent, and it’s been that way for a long time. (2) there are disease areas where a tragedy-of-the-commons situation will mean that controlled clinical trials (which are still the only way to determine if a new drug works) will be much more difficult to run under a “why wait for proof?” system (I have to note that the current bill restricts right-to-try to those patients who are ineligible for clinical trials, which is a good provision). (3) opening up unproven therapies could lead to a moral hazard, where it becomes lucrative to put useless-but-harmless “drug candidates” into “clinical trials” so you can charge desperate patients at “cost”. I mean every quotation mark in that last sentence. I am not the only person who thinks this way. My own suggestion for the last point has already been made: antioxidant phytonutrients – i.e., dried grape juice pills – for Alzheimer’s. Won’t do a thing, but hey, you have the right to try ’em.

There is empirical evidence for all of these positions. Many states have passed their own right-to-try laws (in various forms), and if there has been any decrease in human suffering or advance in medical knowledge through them, I have missed it. To get specific, when Colorado passed its own law in 2014, one of the first companies that announced plans to offer treatments under its provisions was a stem-cell company, Neuralstem. And guess what? None of their treatments ever worked. Most things don’t.

Overall, though, I’m inclined to put this current legislation in the “grandstanding” category. That’s because, as I understand it, there are no provisions that any given company has to provide such an experimental drug for use. It might be better named “right to ask“, and the compassionate-use program already provides just that. What’s changed? Well, now that we have a brand new federal law, all sorts of politicians will be able to talk about how they brought lifesaving therapies directly to critically ill patients, shook up the horrible drug approval process, struck a blow for the common people and stuck it to the drug companies, and on and on. Not always a very instructive spectacle, politics. Scott Gottlieb now gets to implement this law, and good luck to him.


79 comments on “Federal Right to Try”

  1. Danish Khan says:

    Prediction: Mayhem. Untested drugs are as good as poison pills that well…. may or may not work. If the patient’s condition deteriorates, we know where the blame will go. If it gets better, that’s no scientific proof of the drug’s efficacy (n=1). No body wins here.

  2. Isidore says:

    I can’t disagree with your reasoning regarding the utility or, rather, lack thereof of this new law, yet I have trouble reconciling this with my (and many others’) strongly held view that individuals have the right to screw themselves if they so desire without the government preventing them from doing so, as long as by doing so they pose no danger to anyone else (e.g. by failing to become vaccinated against a communicable disease). And while it can be argued that desperate people often do not make rational decisions, it is not the government’s business to stop them (again, as long as they pose no harm to others).

    1. JoeBob says:

      The reconciliation is that if things go sideways with people forcing their way into a trial situation (especially if they were rejected for a regular clinical trial) is that it could endanger the rest of the drug development process, potentially harming the potential patients down the road.

      1. Isidore says:

        Although this argument may hold some truth, it is also the case that many other individual actions have deleterious effects down the road, for example people not watching their diet and no exercising, drinking too much alcohol, etc. all of which affect us all by increasing our medical insurance premiums. But I do not want any government agency coming into my house to tell me what to eat and how much to exercise.

        1. Anoni says:

          Hah, classic conservative talking point. Nice job comrade.

          Before right-to-try, it wasn’t must-take. So why make the argument that without right-to-try big government is going to come in and tell you to hit the treadmill?

        2. Crocodile Chuck says:

          You tell ’em, Isodore!

          They can pry that Twinkie from your cold, dead hand!

          1. Chemist says:

            The problem with liberal elitist snots like you, is you think you will be the one in charge dictating what people can do. How will you feel when someone else is deciding what you can and can’t put in your body? You lack self-awareness.

    2. MTK says:

      Agree with all of this, Isidore.

    3. Derek Lowe says:

      I know what you mean about an individual’s right to screw themselves; I have libertarian sympathies myself. But at the same time, this might turn into a law that should be looked at from another angle, as greatly facilitating unscrupulous people to screw others. I’m not sure how to reconcile that, either, but that viewpoint is what really drives my objections to this sort of legislation.

      1. Mike C says:

        “at cost”

        It wouldn’t address the other issues, but how about if the payments have to be put into escrow until the drug is approved? No approval after X years? Payments have to be returned

      2. loupgarous says:

        Derek, I wonder if “unscrupulous people screwing others” could be prosecuted by other Federal agencies (e.g. Federal Trade Commission, FBI, Postal Inspection Service). Perhaps the single most fraudulent seller of “male performance supplements” was successfully prosecuted and imprisoned by the Federal government, even after Orrin Hatch’s bill protecting the right to take pills in the vain hope of getting bigger, more functional junk passed.

    4. Mark Thorson says:

      That line of reasoning would say Ponzi schemes should be legal. After all, don’t you have the right to try any investment vehicle you want?

    5. regularguy says:

      As Derek pointed out this grants the right to screw yourself only in theory, since drug companys have no obligation to provide their products, and many incentives to refuse.

  3. mal says:

    Who pays for the non approved non-drugs? Who is responsible if there’s harm done due to misuse ? Prediction: for many natural products, there will be companies that make additional profits but little actual medical benefits, but many claims of course. For ethical pharmaceuticals. there will be a lot of apprehension to give drugs free for unapproved indications and a lot of angst on the cost. Plus, there will be lawsuits when the drugs don’t work or harm is caused.

    1. Design Monkey says:

      pays – 1. the person, who wants to try, out of his own pocket, obviously.

      resposibility – 2. the person, who wanted to try, obviously.

      Besides there can not really be lawsuits about harm or inefficencies of unapproved drugs, used under this “right to try” thing. No one promised those “righters-to-try” anything.

      1. Nesprin says:

        I bet that a big insurance co will be sued over coverage of right to try drugs before 2020.

        1. MTK says:


        2. Design Monkey says:

          In any at least minimally just and sensible legal system such lawsuit should be immediately graded as frivolous litigation, plaintiff should be severely punished, and his filing attorney preferably shot immediately on the spot.

        3. UudonRock says:

          I’m sure there will be an attempted lawsuit over issuance refusal to cover drugs not yet FDA approved. There is no real requirement for them to cover all FDA approved drugs as it stands. I can see the lawsuits happening, I can’t see it ending well for the instigator of the suit. An increased chance of legal action could raise premiums for everyone as a result of this.

        4. A Different Scott says:

          No Bet.

      2. M Welinder says:

        > No one promised those “righters-to-try” anything.

        They heard promises, though. Oh boy, did they hear promises.

        1. Anonymous Researcher snaw says:

          Right-to-try is unlikely to accomplish what its backers want. Companies actually trying to make a drug will mostly decline to use right-to-try while snake oil peddlers will abuse it. To quote the Marx Brothers: “You can have any kind of a home you want. You can even get stucco. Oh, how you can get stuck-oh!”

      3. Ken says:

        “resposibility – 2. the person, who wanted to try, obviously”

        Is that written into the law? If not, it’s not obvious, and may not be obvious even after a the first dozen lawsuits.

        Also, if it’s not in the law, that’s another reason for drug developers to be wary about letting people try an unapproved drug.

      4. Anonymous says:

        Design Monkey: “Besides there can not really be lawsuits about harm or inefficencies of unapproved drugs.” While I agree with that, I hope that this law does not protect those perpetrating FRAUD who should still be subject to severe penalties. A biotech with weak but honest data in fruit flies that doesn’t even want to jump into humans is different from snake-oil salesmen promising cures based on smoke and mirrors. Biotechs promoting false and misleading data should be sued out of existence and management prosecuted for criminal fraud (Theranos and others).

  4. b says:

    Any company that runs legitimate clinical trials already has ways to get experimental therapies to those that don’t meet trial selection criteria (expanded access, compassionate use). Data use from these routes is agreed upon with our ultimate judge, the FDA. Why dirty the safety data in a population who is incredibly likely to have adverse events, especially that death one? If an otherwise safe/efficacious drug goes off the rails because of public perception (“this thing is poison, everyone who takes it dies!” or “look, it doesn’t work at all!”), it is indeed a tragedy of the commons and future patients are left to suffer. These companies are only going to distribute experimental drugs through traditional means. This wasn’t a problem to begin with.

    This just means illegitimate companies (not) developing “treatments” are more able to prey on patients when they are at their most desperate.

    1. I had the same takeaway. What does “right-to-try” give you that compassionate use or expanded access programs don’t?

      There are already pathways to get unapproved drugs to patients who need them, so I’m not quite sure why we needed a new law.


    2. Loren says:

      The problem with the existing systems is that adverse events in the compassionate use patients are still reportable. Drug companies are afraid of allowing it because their drug can end up suspected of harm it didn’t do.

      What I would like to see is right-to-try applying to drugs after a successful Phase II trial. I would also like to see a rule that if a patient in a clinical trial appears to obtain benefit from a drug they can continue to use it at cost until such time as the drug becomes generally available. The drug company would not be allowed to say no.

      I would also like to see right-to-try apply to anything that has shown reasonable usefulness anywhere in the world. I’m specifically thinking of the use of viruses as a treatment for bacterial infection. For practical purposes there’s no way to get FDA approval. Isolating exactly what viruses are actually needed is a huge project with **major** ethical problems. (You would normally use the standard therapy for your control–but it’s far more likely the test is inferior to the control than superior to it.)

      1. Anonymous says:

        Re: Loren. (1) Drug continuation before approval if a patient benefits from the clinical trial. Some clinical trials do have that requirement, depending on the disease condition and the treatment and the response… and the ability of the company (e.g., biotech cf., Big Pharma) not to go bankrupt before approval. 🙂

        (Can Pipeline readers help me out? In the 1970s or 80s, a mother accidentally found that a drug approved for another purpose had an unexpected positive affect on her autistic(?) or ADHD(?) son. The drug was discontinued for its intended use by the manufacturer over fears of side affects in pregnant women (I think) and she could not get any more. She told doctors at MGH who initially dismissed her observations. The MDs looked into it further and filed a patent without including the mother, the actual inventor. Ultimately, the drug didn’t work as well as hoped and more research was abandoned. It was big news in the NYT and WSJ back in the 1980s or 1990s. Does anyone remember the drug, the company or any other info? Thank you.)

        (2) Viruses for bacterial infections: Been done, with approval. There is a mechanism in the US to do that. It was the therapy of last resort in this case:

        1. loupgarous says:

          A little Googling turns up Anafranil (clomipramine) as a possibility. FDA only approves it for treatment of obsessive-compusive disorder now. It’s got a lot of adverse events associated with it, most notably serotonin syndrome. Its pregnancy status is C, with AEs observed in neonates of women who’d taken it in late pregnancy.

  5. myma says:

    As someone who worked for a while on the clinical supply side, I always wonder about how the companies are going to supply right to try requests. Its not like there are lots and lots of batches of released for human use sitting around. Each batch has been marked (and not just earmarked, but actually marked) for some purpose. Would one supply from the actual clinical batch – but what if there is an adverse event how would it impact the clinical batch.

    And then someone, somewhere, who requests a right to try is going to equally confound the insurance companies when they asked that it be covered. It is going to happen eventually. Company X offered us the right to try drug, but insurance won’t cover it and the company wants $__0,000 for it, here is a picture of us with our sick kid, all the social media will go nuts, oh those meanies.

    it will be interesting to see how it plays out to say the least.

  6. luysii says:

    Let’s not forget snake venom for ALS and MS, distributed by a fraudster in Germany — you had to stay in the motel he owned to get it. Patients would return and say they saw “remarkable things” but they didn’t get better

  7. me says:


  8. MoMo says:

    Ye Gods! The snake-pit has opened!

  9. tlp says:

    While I agree with a spirit of opposing right to try, I’m not sure if grape juice pills for Alzheimer is a good argument here. AFAIU from the post, there must be a legit trial ongoing so that population of ineligible patients can exist. That by itself should be a high enough financial burden for sham treatments. After all their model doesn’t include any trials at all.

  10. Onlooker says:

    Will the Right- to-Try legislation open up new avenues for the Right -to-Die ?
    Could Terminal patients “try” a lethal dose of an experimental drug under these new guidelines ?

  11. shanedorf says:

    Will the Right- to-Try legislation open up new avenues for the Right -to-Die ?
    Could Terminal patients “try” a lethal dose of an experimental drug under these new guidelines ?

  12. Eugene Fisher says:

    This is frought with un-intended consequences. Just got training on the EU privacy law and my first thought was that every IT provider in the EU is going to grind to a halt over the flood of requests from people wanting to review their personal data. I suppose pharmaceutical companies could isolate right to try patients results from regular clinical trial results but the temptation will also be there to try to some how re-habilitate that data and use it, especially if it has enough outliers. The scammers and fraudsters have been around forever (Laetrile). The supplement industry already gets what is essentially a free pass from the FDA with only passing enforcement. Homeopathic “medicines” (Airborne) are on the shelf in CVS alongside all the other OTC medicines that actually have some efficacy and make claims in bold lettering alongside the warning (in small type) that these claims have not been evaluated by the FDA. This seems to circle back to the level of critical thinking that most ordinary people use in there daily life, which is abysmally low.

    1. fajensen says:

      …. was that every IT provider in the EU is going to grind to a halt over the flood of requests from people wanting to review their personal data …
      Deservedly so, because they are a bunch of plonkers and cheap-skates the lot of them!

      They have had “years” to get rid of toxic data-dumps and also years to write a “GDPR Compliance API” so that the process is automated.

      As it is, someone will rapidly corner the market by offering GDPR compliant CRM services and companies will subscibe to this, like already happened with accounting when people suddenly had to interface with electronic billing and EDI.

  13. Crocodile Chuck says:


    Don’t tell Alex Tabarrok!


  14. Serious researcher says:

    Oh, poor little drug companies and entrepreneurial professors, no more monopoly on ability to try medicine if you are sick. Big bad business republicans really did it this time. Grow up.

    1. Design Monkey says:

      Obviously you are too stupid to understand a couple of little nice nuances, that that bill has.
      1. who should pay for those “wanters to try” whims? In true conservative republicanic spirit it should be wanter itself. He wants, he pays fully himself for his wants. Nobody else. Trying to push insurance companies to pay for unproven methods or making pharmaceutical company give away their product for free would be typical red commie approach. Verboten for likes of you.
      And you know, there may be a lot of wanters, but there will be just a tiny little of payers, if they will have to pay a full price.
      2. When drug is still in trials phase, there isn’ t anything like an established production line for it. Typically it’s made as specialty batches precisely for trial puposes in specialty GMP pilot plant, at huge prices. And there just isn’ t any spare drug floating around to be given away or even sold to various side wanters . Playstation sold out at Christmas. Come back later with your wants. Possibly a couple years later, when drug will be approved. Mandating pharmacetical companies to compulsory produce and distribute extra drug, again would be red commie methods. Verboten for you.

      So, actually that your tryers bill is just another hollow populistic bullshit. And your own Gottlieb will be one, who will have to try to make a cake or at at least a not too burned bun from that trumpie pile of absolute crap. Not likely that he will succeed.

      1. Lola says:

        Haha…fantastic response Design Monkey. Of course, we’re assuming ‘right-to-tryers’ are hardened right wing libertarians, which sounds a reasonable assumption. But then you’ve got the proportion who think emotionally (only). They can’t be helped – their version of logic is non-existent, and can make fairy-dust out of horse-shit if it ‘feels good/right’. Interestingly, they are the deliberate target of the drug company’s supposedly ‘independent’ patient groups – people so caught up in the emotion of their condition, there is no point arguing. While I’m no ‘big-pharma’ conspiracist (and find these people utterly useless in advancing science or progressing anything at all), it is a cynical play on the drug company’s behalf to get these people behind their marketing efforts. The mark of these people is ‘n=1=proof’!

        1. Chemist says:

          “Wtf I hate having the right to decide what to put in my body now” – Libtards

          1. Design Monkey says:

            You can put whatever snake oil you want in your body, as far as you pay for it yourself, trumpie-tard fake “chemist”. 1. you won’t be so happy about prices. trumpie farts are cheap, yout rightie-try fake “drugs” will not be 2. in unregulated medicines market significant part of population would be either outright poisoned by untested toxic “medicines” or swindled out of money by useless non-working ones. Both outcomes generally are regarded as not especially desirable, but are actually good, when applied to to trumpie-tards. Just don’t run around screaming mommy, when you will get, what you yourself wanted.

    2. BadChemist says:

      You’ve always had the right to ingest whatever non-scheduled substance your dumb-a** wanted to if you had the knowhow to make it. Don’t expect anyone to jump at the chance to sell you what could be poison.

      1. Seriously says:

        Yeah, ive of course thought of that and its bogus because who is going to try a medicine that has not gone through some federal oversight…none. the point of the law is to establish a pathway to try a possibly* legitimate medication as a last chance effort that has gone through a cursory review by good scientists.

        1. Design Monkey says:

          >because who is going to try a medicine that has not gone through some federal oversight…none.

          You blatantly underestimate levels of idiocy, that are present in human population.

        2. loupgarous says:

          Even medications that have been awarded New Drug Approval can have problematic toxicity undiscovered during clinical trials, Vioxx and several of the fluoroquinolone antibiotics notably among them. In the case of Right to Try, we’re talking about drugs and therapies which haven’t even had that degree of pharmacovigilance.

          But Right to Try insulates manufacturers from liabilities, which is very troubling given toxicity of things like odanacatib never showed up until Phase III trials. Right to Try patients wind up being unwitting tox subjects with no Institutional Review Boards protecting their interests or controlling the way in which they get unapproved drugs. A “fact sheet” produced by the Goldwater Institute actually describes delays associated with IRBs as one of the reasons why the FDA’s compassionate use wasn’t fast enough and Right to Try (then, only at the state level) was necessary. Of course, they don’t go into the reason IRB approval takes time – because it’s not a “rubber stamp” for the manufacturers or FDA, but an independent review process intended to protect the patient.

          Quoting another document by the Morgan Lewis law firm:

          “Eligible investigational drugs that are provided to eligible patients are exempt from requirements for FDA approval, INDs and most of the corresponding IND regulations, certain labeling requirements including adequate directions for use, and FDA’s regulations on informed consent and institutional review board (IRB) approval.”

          In Right to Try, patients have no legal recourse against manufacturers of the drugs patients get. Manufacturers thus have no real incentive to minimize risk of unforeseen toxicity in those drugs. And as the quote above shows, even the protective measures of the FDA’s Investigational New Drug regulations are waived.

          You don’t have to go all the way to Snidely Whiplash Pharmaceuticals territory to see how desperate patients and their loved ones can get royally screwed during and after treatment under Right to Try. But Big Press let it pass, when all of these issue could and should have been raised publicly.

  15. PI tips says:

    Now and then i give a student a whoopin if they dont come in on holidays. It keeps them honest. Unlike you suckers, all my grad students will be at work on Monday.

  16. PI tips says:

    A good line from a recent film was ” i am never going to be president “. Most of the golden childs that frequent these types of power blogs should admit the same. Sure, your life will be less fun, but everyone around you will thank you.

  17. Tourettes of Chemistry says:

    Just one small step from bio-hacker to pharma-hacker.

    World class instructive dosing information:

    That patient likely saw ‘remarkable things’ as alluded to above.

    Experiment design with side-by-side bathtubs in the garage or on the porch in conjunction with self-reported patient comfort, efficacy and safety endpoints will ensure meaningful outcomes in determining gender-based dosing parameters for the entire global population.

    Toilet paper and modulators of physiology should of course be marketed in identical fashion. By extrapolation, invention and discovery must be identical exercises as well.

    1. mjs says:

      At the other extreme from the paper you mention “Lysergic Acid Diethylamide: Its Effects on a Male Asiatic Elephant” is one on “LYSERGIC ACID DIETHYLAMIDE – MUTAGENIC EFFECTS IN DROSOPHILA” LS Browning, Science 161 (1968) 1022. What caught my attention back then was the method: “The testes of 75 virgin 6-day-old males…were bathed (by intraperitoneal injection) in approximately 0.3 microliters of a fresh solution…” Very high dose per unit body weight, but startling technique.

  18. Anonymous says:

    Did the recent death of Aaron Traywick on April 29 show up in Pipeline? Q.v., and wikipedia. He was surely an example of a Right to Try-er, except he was trying stuff that he made on his own; no Big Pharma or FDA for him.

    Traywick was a DIY biohacker with no formal scientific or medical training. From The Atlantic, his “goal was to develop and test new gene therapies without the expense and rigor of clinical trials or the oversight of the FDA. Traywick wanted to cure cancer, herpes, HIV, and even aging, and he wanted to do it without having to deal with the rules and safety precautions of regulators and industry standards.”

    I would like to find cures for all of those diseases, and others, too!

    His death may have been accidental and unrelated to his self-experimentation. Autopsy results won’t be available for several more weeks.

    I think his choice of self experimentation was legal, other than for the possible use of commercial reagents labeled “not for human use” or similar.

    1. Bill says:

      A stupid argument, no one is going to do what he did because its irrational. What is rational is trying drugs that have had a cursory review by serious scientists as a last chance for your dying. The cold heart of yours is annoying.

      1. Some idiot says:

        Bill, with all respect, I don’t think anyone here on this blog is being cold/hearted. I think almost all on this blog are actually genuinely interested in trying to find new medicines that will help people.

        I really do understand your point, and taken completely in isolation, I can understand the desire of someone who is terminally ill and has tried everything that is currently available, and would like other opportunity to try something that has just entered clinical trials and
        “looks promising”. But this where the problems start…

        “Looks promising” is a problem in itself… Pre-clinical? Forget it. End of Phase one? Forget it. It has just proven that it doesn’t kill people immediately. End of phase 2? Nope. Interesting, but not promising.

        And then you have to look at the risk of results from “right to try” people muddying the waters of the true trials, making proper evaluation of the results even harder.

        Plus all the tricky logistical nuts and bolts questions such as to “which batch” (as mentioned earlier), and the potential loopholes that this can create for swindlers to milk terminally ill people for their money. Yes, in principle it should be very straight forward, but in practice it is a lot muddier than that.

        So no, we are not cold/hearted, just very aware of the problems that dwell behind it. I take strength from a very dear friend of mine who died from cancer (she was also a scientist). When it became clear that treatment was no longer an option, she contacted every research group she was aware of to say that she was available for the testing of drugs or protocols. Not because she believed that she would be cured, but because she wanted to make sure that society and science could get the most out of her situation.

        1. mark antell says:

          Some idiot says, “I really do understand your point, and taken completely in isolation, I can understand the desire of someone who is terminally ill and has tried everything that is currently available, and would like other opportunity to try something that has just entered clinical trials and “looks promising”. But this where the problems start…”

          I think, yes you do understand the point well enough to argue … but your arguments come from a cold heart. You would prevent desperately ill persons from a taking chance to save themselves. Shame.

          1. Shazbot says:

            Many of these aren’t simply ‘taking chances to save themselves’. It’s the right to end their lives in painful misery alone in rooms looked after people who don’t care and are there to extract money from the people who are simply wanting hope. There are plenty of people willing to sell you expensive, noisy machinery, hook you up with enough salt solution to have you dying of thirst while peeing every hour, happily encourage you to sell everything you own so you can die in a empty room once they’re done sucking out your empty husk and leaving you in immense pain.

            With treatments that can work and are effective, sometimes it isn’t the right thing to accept a course of treatment. With treatments that do not work, this is much more often the correct thing to do.. live your life as best you can, and make informed choices, not be subject by the latest quack who is willing to trade false hope for filthy luchre.

          2. Some idiot says:

            I am sorry to hear that you feel that way, but after reading my post in its entirety, I am really, seriously, curious and completely not understanding why you say that my “arguments come from a cold heart”. What is it about my comments, coming from a position of many years practical experience, outlining why this new law is likely to be worse than useless, makes you say that I have a cold heart? Or is it simply because anyone who disagrees with you necessarily has a cold heart?

            Not to mention the personal anguish and pain I have had when family members or very close and dear friends have died due to conditions that could not be treated…? The reason I went into the research and development of new medicines was precisely to try and help people who cannot currently be treated. They cannot be treated with my heartbreak and anguish, so instead I try to use my logic and experience instead, to be a part of creating treatments that have been scientifically shown to be useful. In my book this is better than letting them drift into the hands of snake-oil salesmen with easy promises of fake cures. It is (unfortunately) these snake-oil salesmen who will be the only real beneficiaries of this new law.

        2. loupgarous says:

          Pretty much what I figured to do when I signed up for clinical trials of PRRT – the standard of care to that point, octreotide depot injections, wasn’t halting progression of my cancer, and the largest solid metastases were in my liver, hence inoperable. So I rolled the dice, thinking “negative results are still results, and at least we’d find out what didn’t work.” Of course, I wasn’t as detached as that implies… just hoping the odds were in my favor. And PRRT worked.

      2. Anonymous says:

        I don’t think I argued for or against any side of the issue. My post was mostly statements of news or facts. Traywick died; he tried his own concoction; he ignored FDA warnings; I would like to cure diseases; the cause of death is still unknown; self-medicating is legal; depending on how you acquire some reagents, it may be illegal to apply them for human use. (For example, Tecfidera is dimethyl fumarate for MS. It is $55,000 per year. 240 mg, twice per day is 176 g per year. A 250 g bottle from SIAL is around $100 – but it is not for human use.)

        Like you, many biohackers considered Traywick’s actions to be irrational and harmful to the biohacking effort.

        Which “side” did you say I was on?

  19. Massively accomplished says:

    Can we make a max word length for chemists on blogs? I get it with papers, you have to write a bunch fill up space for the journal, but the first rule of writting well is reduce the word count and there is no need for verbosity on blogs. In fact, it is actually what you dont want. It really suggests to me that you all like to hear yourselves talk.

    1. Some idiot says:

      In general on this blog, my impression is that the length of the blog here (and the comments) are pretty proportional to the intellectual content.

      By that metric, however, your comment is around 69 words too long.

  20. BostonChemist says:

    Looks like Vertex has a lot of postings for chemists.
    Guess they were just flushing out the dead wood with recent departures.
    Novartis seems to be the bottom of the heap in Boston these days.

  21. mjs says:

    At the other extreme from the paper you mention “Lysergic Acid Diethylamide: Its Effects on a Male Asiatic Elephant” is one on “LYSERGIC ACID DIETHYLAMIDE – MUTAGENIC EFFECTS IN DROSOPHILA” LS Browning, Science 161 (1968) 1022. What caught my attention back then was the method: “The testes of 75 virgin 6-day-old males…were bathed (by intraperitoneal injection) in approximately 0.3 microliters of a fresh solution…” Very high dose per unit body weight, but startling technique.

  22. Joey says:

    It is their life or death. Let them decide, not you.

    1. Lola says:

      It’s your kind of naive, simplistic thinking that characterises the over-emotional from the genuinely caring. Just one example: many of those people who are doing their own thing with ‘right-to-try’ substances will require (and get) palliative care that will be far more expensive that it would have otherwise been ie. a dollar spent on their care not availble for someone else. All to indulge their need to try something not fully tested. And that’s just one side effect, there are numerous others. The main point is: ‘right-to-try’ laws cannot be evaluated based on emotive criteria only – rather many complex arguments require dissection in order to do this properly. And this is an anathema (or just plain too hard to understand) for those that reduce the problem to ‘my life – my choice’. (Who’s going to pay’ is another issue – covered by other postings here though).

  23. David Edwards says:

    One word. Thalidomide.

    Which is possibly the canonical example of a drug that was propelled through the standard route, only for the standard route to be found wanting when tracking down its manifest deleterious effects upon the foetus when taken by pregnant women. That was a wake up call for the entire industry ultimately spanning 25 years and numerous lawsuits.

    Benoaxprofen was another example – a drug for managing arthritis symptoms, that ended up killing too many of the patients taking it. Eli Lilly’s reputation in the UK in particular was immolated with a flamethrower over that one.

    Practolol caught the then ICI Pharmaceuticals division on the hop, after it was found to have a raft of side effects not observed in animal tests.

    Quite simply, if a drug company can be caught with its pants down, even if it jumps through all the regulatory hoops, and exercises demonstrable diligence with respect to weeding out rogue medications, despite having the benefits of billion-dollar R&D budgets and small armies of experts overseeing the development at every step, then the scope for even more disastrous outcomes can only be widened by this latest push for “right to try”. That widening of scope will take place even if all the participants in the endeavour exercise scrupulous honesty throughout, but of course, one doesn’t have to look far to find examples in the industry of, let’s call it ‘failure to attain the standard’ here, just in case the unnamed guilty suspects are tempted to respond aggressively to a no-nonsense description of duplicity.

    In an endeavour that involves risk or hazard to human life, one should be looking for proper methods for reinforcing rigour of practice, not dynamiting a large hole in the side thereof. “Right to try” strikes me as a particularly egregious example of said dynamiting, because it’s practically tailor-made for the spawning of more Martin Shrkelis – sleazeball sharks and douchebags out to make fat wads of cash by exploiting desperate and vulnerable people, and who will all be rejoicing at the prospect of their ruthless avarice being given the green light. Who in turn, via their mendacious activities, will poison the well for the legitimate participants. At the moment, there’s still enough people who recognise the distinction between the hard graft of the actual scientists, and the dick-swinging power games of the C-suite MBAs, but this measure might damage even that last reserve of goodwill.

    1. Chris Phoenix says:

      Literally every endeavor involves risk or hazard to human life, if you chase the consequences far enough.

      Medicine in the U.S. (not just medical research, but that’s part of it) has been drowned in regulations – it is actually illegal for a non-doctor to buy a bag of medical saline solution, one of the most harmless objects I can imagine!

      Attempts to fix the problem piecemeal may well make it worse. I don’t know the current legislation’s details and implications well enough to argue for or against it (and neither, I suspect, do the lawmakers or the readers of this blog).

      Some regulation is necessary – I’m not arguing for none. But surely the collection of regulations we have right now is too much in many areas (while, plausibly, not enough in a few areas).

      A blanket argument of “It’s risky, so we need more regulation” just won’t fly with any medical consumer who sees the disastrous state of U.S. health care today. People get bankrupted, and die, for lack of services that are simply too costly to afford. Our infant mortality statistics alone, compared with other countries that spend a lot less on health care, should be a wakeup call.

      We’re doing things fundamentally wrong here, and broad arguments for more regulation are not a useful way to attack attempts to fix the problem.

      1. Lola says:

        “We’re doing things fundamentally wrong here, and broad arguments for more regulation are not a useful way to attack attempts to fix the problem.”
        This may well be true, but is so vague and motherhoody that it bears little relevance to ‘right-to-try’ legislation. Moreover, the problems with the US health system can hardly be reduced to ‘too much regulation’. The disgrace that is the US health system is borne of a myriad of problems: some historical, but in my view, most cultural with an entire society’s aversion to anything that reeks of ‘socialisation’ – on all sides: patients, doctors, and insurers. I see the insurers and the doctors cleaning up however, so the cost is borne exclusively by the patients. Empirically, health systems that work are fundamentally oriented around the ‘social’, not the ‘individual’ and this is the biggest barrier to fixing America’s intractable health system problems. Spending more per capita, and getting worse results is the outcome – a failure in anyone’s book, regardless of your point of view.

    2. Emjeff says:

      What does ANY of this have to do with Right-to-Try? Adverse events are seen in all clinical trials. As well, what people are forgetting is who this actually affects – the drug companies who are actually trying to study a real medicine. The hucksters are already selling their crap – this does not affect them in the slightest, since they don’t really worry about the FD&C Act anyway.

      The only thing this law will do is streamline the ability to try a medicine in people who are in desperate situations. This will not enrich anybody, since, as pointed out before, the hucksters and thieves don’t worry about science and the law anyway.

  24. Scott says:

    So Derick, your statistics 90% of experimental drugs don’t work. We try them anyway. why is that? Because we hope they work.

    In the future, 90% of experimental drugs won’t work. But you don’t want to hear from people who thing nothing will really change? What could change? You suspect 95% of experimental drugs won’t work? And this would be worth shouting from the rooftops about? Are you insane or did you fail high school statistics?

    1. Chemist says:

      This is just liberal cognitive dissonance on display. Nobody wants to give Trump credit for trying new things that could potentially help people. Hence all the “wtf I hate having the right to decide what goes in my body now” posts.

  25. David Young MD says:

    What about the “right to try” approved drugs that are not approved for your cancer? These drugs have a much higher chance of helping. For any given cancer there is probably an approved drug with a 50-50 chance of helping…. but the drug is approved for another cancer and not yours… and therefore would not be paid for.

    The “right to try” people have gone after the wrong set of drugs to request availability.

    I would say, however, that all of the immune checkpoint inhibitor drugs have been made available free to us oncologists for any cancer where they are not approved. This has been a tremendous breakthrough for people. It is unprecedented in the history of oncology and my gracious thanks for the pharmaceutical industry for doing this.

  26. Anon grammar lint says:

    Derek, you have a dangling clause : “(As an earlier link shows”. Not sure if you wanted an internal bookmark, the same link repeated, or just a closing parathesis.

    1. Derek Lowe says:

      Ack – that must have been left over from the composition process. Thanks!

  27. Anonymous says:

    Dear Derek,
    The “right-to-try” legislation signed into law today does not give terminally-ill patients the right to test on themselves any ineffective and/or possibly toxic chemical(s). They always had that right and the FDA does not regulate suicide attempts, nature does.

    It does not give them the right to try already marketed products for a different indication… licensed physicians already have that right and can prescribe any marketed drug they believe would benefit their patients for any illness, regardless of the FDA-sanctioned therapeutic use. Insurance companies decide whether to cover or not the cost of an unapproved treatment.

    What the “right-to-try” law does, is remove the FDA from the interaction between the patient, his/her physician and the manufacturer of an experimental treatment currently being developed under an IND or NDA/BLA, past Phase 1, not on clinical hold but not yet approved for marketing.

    Prior to the signature into law of the Right-to-try Act of 2017, a patient who believed he/she could benefit from such product was required to have the attending physician petition the manufacturing drug company for supply, and then petition the FDA for a “compassionate use” of the product. Such requests were granted in the majority (99%) of the cases, and when urgent, immediately over the phone. The FDA keeps a list of medical officers available to respond to emergency IND requests 24/7. Treatment could begin immediately after the verbal agreement but did require follow-up with a written form FDA 3926 justifying the use of the unapproved product. The request was assigned an IND number, and the drug supplier could use this number to ship the product to the requesting physician, since current laws prohibit the transport of experimental drugs across state lines without FDA approval. From now on, the attending physician will deal only with the experimental drug supplier without interaction from the FDA.

    While this new approach may save physicians a phone call and an electronic submission, its impact on the enthusiasm of pharmaceutical companies for such “compassionate use” is unclear. As other have already pointed, drug supply is rarely plentiful in phase 2/3, and the manufacturer may have to prioritize between a compassionate gift and data that would contribute to drug approval. Because such requests can only be made by patients with life-threatening diseases, the likelihood of an unfortunate outcome, regardless of the treatment, is not negligible. Such occurrences can lead to uncomfortable discussions with the FDA review team when the FAERS report arrives, and few sponsors seem to enjoy delays in the approval of billion-dollar drugs. In the prior system, the division responsible for the product’s review was also approving the compassionate use, and one could expect at least some familiarity with the circumstances surrounding the compassionate treatment when the outcome was less than optimal… This will not be the case anymore. Instead, sponsors will have annual reporting requirements to FDA regarding their use of drugs under Right to Try, including any adverse event they encountered.

    Fortunately, as many of the readers of this blog can personally attest, the pharmaceutical industry is well known for its altruistic tendencies, and mere financial and risk considerations are unlikely to prevent the generous gifting of expensive experimental products for indications widely outside of the known pharmacology. Hence, after today, patients will enjoy access to new promising treatment options completely unanticipated by the medicinal chemists, biologists and pharmacologists who designed the molecule, but instead imagined by their attending physician. Rational drug design is out, replace by “random stuff on random condition”… but as our White House says “it doesn’t matter, he’s dying anyway”

    I would like to take this opportunity to thank Derek and his viewers for many years of enlightening posts, and for the synthesis of so many compounds with such interesting properties. Keep that pipeline flowing!!!

    1. Phil says:

      I (UK, not a chemist) dearly love this comment which I believe has attained a 100% irony score even on the Waugh scale. Agree with last paragraph.

      1. sgcox says:

        Second to that. Whoever is this Anonymous, I would love to see him to comment more often.

  28. lola says:

    Unfortunately, these ‘right-to-try’ laws are borne of our current ‘Google Generation’. An ordinary joe can do 20 minutes research on the internet, read a few articles (many of which will come from the profiteers) and know as much as the specialist who’s trained for 15 years. The thing missing is the clinical golden rule: what looks good on paper may not work in reality. So the gullible/naive/desperate educate themselves on things like the mechanisms of a pathology, read about a pathway (for example) that somehow ‘blocks’ a bad situation, and assume the human body will behave they way it should on paper. Ethical clinicians know this is not how medicine works – until it’s control trialled what works on paper is at best a hopeful hypothesis. But..this is an extremely difficult proposition to sell to the aforementioned gullible/naive/desperate. Hence the demand for ‘right-to-try’. There’s no solution to this dilemma, and we should stop looking for one…rather expect a messy road as untested remedies kill/maim and occasionally cure. (An acid test for those committed to ‘right-to-try’ – surely they would support legalisation of ALL drugs – after all, it’s a user’s right to put whatever they want into their bodies right?). If ‘right-to-try’ laws were combined with a compulsory statement that e.g. ‘90%’ of ‘right-to-try’ treatments fail (based on real evidence to date), the laws would be acceptable. I don’t know, I think it’s one of those things we need the dignity to accept we can’t solve.

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